WO2018049009A3 - Modulation des gènes du foie - Google Patents

Modulation des gènes du foie Download PDF

Info

Publication number
WO2018049009A3
WO2018049009A3 PCT/US2017/050446 US2017050446W WO2018049009A3 WO 2018049009 A3 WO2018049009 A3 WO 2018049009A3 US 2017050446 W US2017050446 W US 2017050446W WO 2018049009 A3 WO2018049009 A3 WO 2018049009A3
Authority
WO
WIPO (PCT)
Prior art keywords
modulation
liver
liver genes
genes
klkb1
Prior art date
Application number
PCT/US2017/050446
Other languages
English (en)
Other versions
WO2018049009A2 (fr
Inventor
Anthony Conway
Gary K. Lee
David Paschon
Lei Zhang
Original Assignee
Sangamo Therapeutics, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sangamo Therapeutics, Inc. filed Critical Sangamo Therapeutics, Inc.
Priority to AU2017324462A priority Critical patent/AU2017324462B2/en
Priority to CA3035534A priority patent/CA3035534A1/fr
Priority to JP2019512750A priority patent/JP7256739B2/ja
Priority to EP17849517.2A priority patent/EP3509645A4/fr
Publication of WO2018049009A2 publication Critical patent/WO2018049009A2/fr
Publication of WO2018049009A3 publication Critical patent/WO2018049009A3/fr
Priority to JP2023057789A priority patent/JP2023082133A/ja

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10—Antioedematous agents; Diuretics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121—Serpins
    • C07K14/8125—Alpha-1-antitrypsin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • C12N15/8616—Special methods for targeting systems
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/867—Retroviral vectors
    • C12N15/8676—Special methods for targeting systems
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0006—Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6445—Kallikreins (3.4.21.34; 3.4.21.35)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Urology & Nephrology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

L'invention concerne des compositions et des procédés de modulation de l'expression génique dans le foie notamment la modulation de PCSK9, TTR, SERPINA1, KLKB1 et/ou HAO1.
PCT/US2017/050446 2016-09-07 2017-09-07 Modulation des gènes du foie WO2018049009A2 (fr)

Priority Applications (5)

Application Number Priority Date Filing Date Title
AU2017324462A AU2017324462B2 (en) 2016-09-07 2017-09-07 Modulation of liver genes
CA3035534A CA3035534A1 (fr) 2016-09-07 2017-09-07 Modulation des genes du foie
JP2019512750A JP7256739B2 (ja) 2016-09-07 2017-09-07 肝臓遺伝子のモジュレーション
EP17849517.2A EP3509645A4 (fr) 2016-09-07 2017-09-07 Modulation des gènes du foie
JP2023057789A JP2023082133A (ja) 2016-09-07 2023-03-31 肝臓遺伝子のモジュレーション

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662384428P 2016-09-07 2016-09-07
US62/384,428 2016-09-07

Publications (2)

Publication Number Publication Date
WO2018049009A2 WO2018049009A2 (fr) 2018-03-15
WO2018049009A3 true WO2018049009A3 (fr) 2018-04-19

Family

ID=61281860

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2017/050446 WO2018049009A2 (fr) 2016-09-07 2017-09-07 Modulation des gènes du foie

Country Status (6)

Country Link
US (2) US10960085B2 (fr)
EP (1) EP3509645A4 (fr)
JP (2) JP7256739B2 (fr)
AU (1) AU2017324462B2 (fr)
CA (1) CA3035534A1 (fr)
WO (1) WO2018049009A2 (fr)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL266862B2 (en) 2016-12-01 2024-01-01 Sangamo Therapeutics Inc Tau modulators and methods and preparations for their administration
CA3061612A1 (fr) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Nouveaux lipides carbonyles et formulations nanoparticulaires lipidiques pour l'administration d'acides nucleiques
WO2020010186A1 (fr) * 2018-07-06 2020-01-09 Derek Klarin Variants de pcsk9
BR112021001546A2 (pt) * 2018-07-31 2021-05-04 Intellia Therapeutics, Inc. composições e métodos para a edição de gene do hidroxiácido oxidase 1 (hao1) para o tratamento da hiperoxalúria primária tipo 1 (ph1)
WO2020072684A1 (fr) * 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Modulateurs génétiques modifiés
WO2020132659A1 (fr) * 2018-12-21 2020-06-25 Precision Biosciences, Inc. Modification génétique du gène de l'hydroxyacide oxydase 1 pour le traitement de l'hyperoxalurie primaire
US11453639B2 (en) 2019-01-11 2022-09-27 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
EP3801011A1 (fr) 2019-06-04 2021-04-14 Regeneron Pharmaceuticals, Inc. Animaux non humains comprenant un locus ttr humanisé ayant une mutation bêta-slip et procédés d'utilisation
WO2021158883A1 (fr) * 2020-02-07 2021-08-12 Regeneron Pharmaceuticals, Inc. Animaux non humains comprenant un locus klkb1 humanisé et procédés d'utilisation
JP2023535365A (ja) 2020-07-16 2023-08-17 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子に使用するためのカチオン性脂質
CA3172481A1 (fr) 2020-11-12 2022-05-19 Precision Biosciences, Inc. Meganucleases modifiees ayant une specificite pour les sequences de reconnaissance du gene de la dystrophine
US20230034581A1 (en) 2021-06-04 2023-02-02 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
WO2023173072A1 (fr) * 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Systèmes et procédés de modulation génétique pour traiter une maladie hépatique
TW202405175A (zh) 2022-06-07 2024-02-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100113575A1 (en) * 2007-03-27 2010-05-06 Ayesha Sitlani Method for detecting autoprocessed, secreted pcsk9
US20140356958A1 (en) * 2012-12-17 2014-12-04 President And Fellows Of Harvard College RNA-Guided Human Genome Engineering
US20150128309A1 (en) * 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal Maize Loci
US20150329875A1 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
US20150335708A1 (en) * 2014-05-08 2015-11-26 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
US20160153005A1 (en) * 2013-06-17 2016-06-02 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy

Family Cites Families (83)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2181548C (fr) 1994-01-18 2009-11-03 Carlos F. Barbas, Iii Derives de proteine a doigts zinciques et procedes associes
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE45795E1 (en) 1994-08-20 2015-11-10 Gendaq, Ltd. Binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
AU746454B2 (en) 1998-03-02 2002-05-02 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
ATE309536T1 (de) 1999-12-06 2005-11-15 Sangamo Biosciences Inc Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
WO2001059450A2 (fr) 2000-02-08 2001-08-16 Sangamo Biosciences, Inc. Cellules destinees a la decouverte de medicaments
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
CA2407460C (fr) 2000-04-28 2013-06-25 Sangamo Biosciences, Inc. Modification ciblee de la structure de chromatine
WO2001083819A2 (fr) 2000-04-28 2001-11-08 Sangamo Biosciences, Inc. Procédés d'élaboration de molécules régulatrices exogènes
US7029847B2 (en) 2000-05-16 2006-04-18 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
WO2002026959A2 (fr) 2000-09-28 2002-04-04 Sangamo Biosciences, Inc. Reprogrammation nucleaire au moyen de iwsi et atpases de remodelage de la chromatine associees
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
CA2474486C (fr) 2002-01-23 2013-05-14 The University Of Utah Research Foundation Mutagenese chromosomique ciblee au moyen de nucleases en doigt a zinc
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP2806025B1 (fr) 2002-09-05 2019-04-03 California Institute of Technology Utilisation de nucléases à doigt de zinc pour stimuler le ciblage de gènes
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2005233550B2 (en) 2004-04-08 2010-11-18 Sangamo Therapeutics, Inc. Treatment of neuropathic pain with zinc finger proteins
CA2579677A1 (fr) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Production de proteines: compositions et methodes
WO2007139898A2 (fr) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Demi-domaines résultant de clivage par génie génétique
WO2007139982A2 (fr) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Procédés et compositions pour l'inactivation de gènes
CN101679977B (zh) 2007-04-26 2013-05-01 桑格摩生物科学股份有限公司 向ppp1r12c基因座的靶向整合
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2281050B1 (fr) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Constructions donneuses linéaires pour une intégration ciblée
EP4154903A1 (fr) 2008-04-22 2023-03-29 Vib Vzw Éléments régulateurs d'acides nucléiques spécifiques au foie et procédés et leur utilisation
CN102159722B (zh) 2008-08-22 2014-09-03 桑格摩生物科学股份有限公司 用于靶向单链切割和靶向整合的方法和组合物
KR101803737B1 (ko) * 2008-12-04 2017-12-01 상가모 테라퓨틱스, 인코포레이티드 징크 핑거 뉴클레아제를 이용한 랫트의 유전체 편집
EP2206723A1 (fr) 2009-01-12 2010-07-14 Bonas, Ulla Domaines modulaires de liaison à l'ADN
US9234016B2 (en) * 2009-07-28 2016-01-12 Sangamo Biosciences, Inc. Engineered zinc finger proteins for treating trinucleotide repeat disorders
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
SG181601A1 (en) 2009-12-10 2012-07-30 Univ Minnesota Tal effector-mediated dna modification
CA2788560A1 (fr) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Demi-domaines de clivage techniques
US9255259B2 (en) 2010-02-09 2016-02-09 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
CA2796600C (fr) * 2010-04-26 2019-08-13 Sangamo Biosciences, Inc. Edition du genome d'un locus de rosa en utilisant des nucleases a doigt de zinc
EP2571512B1 (fr) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Nouvelles protéines se liant à l'adn et leurs utilisations
AU2011281062B2 (en) 2010-07-21 2015-01-22 Board Of Regents, The University Of Texas System Methods and compositions for modification of a HLA locus
CA2814143C (fr) 2010-10-12 2020-09-08 The Children's Hospital Of Philadelphia Procedes et compositions pour traiter l'hemophilie b
CN103917644A (zh) 2011-09-21 2014-07-09 桑格摩生物科学股份有限公司 调控转基因表达的方法和组合物
EP2771457B1 (fr) 2011-10-27 2017-11-22 Sangamo Therapeutics, Inc. Procédés et compositions pour la modification du locus hprt
CA2854819C (fr) 2011-11-16 2022-07-19 Sangamo Biosciences, Inc. Proteines de liaison d'adn modifiees et utilisations de celles-ci
JP6490426B2 (ja) * 2012-02-29 2019-03-27 サンガモ セラピューティクス， インコーポレイテッド ハンチントン病を治療するための方法および組成物
RU2650819C2 (ru) * 2012-05-07 2018-04-17 Сангамо Терапьютикс, Инк. Способы и композиции для опосредованной нуклеазой направленной интеграции трансгенов
HUE041553T2 (hu) 2012-07-11 2019-05-28 Sangamo Therapeutics Inc Lizoszomális tárolási betegségek (LSD) kezelése és génterápia
ES2812599T3 (es) 2012-08-29 2021-03-17 Sangamo Therapeutics Inc Procedimientos y composiciones para el tratamiento de una afección genética
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP4234696A3 (fr) 2012-12-12 2023-09-06 The Broad Institute Inc. Systèmes de composants crispr-cas, procédés et compositions pour la manipulation de séquence
CN105263312A (zh) * 2013-04-05 2016-01-20 美国陶氏益农公司 用于在植物基因组内整合外源序列的方法和组合物
CA2910489A1 (fr) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Procedes et compositions pour le traitement d'une maladie genetique
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
CN112263682A (zh) * 2013-06-27 2021-01-26 罗氏创新中心哥本哈根有限公司 靶向pcsk9的反义寡聚体和缀合物
CA2920899C (fr) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions de liaison de domaines de liaison a l'adn et de domaines de clivage
WO2015066634A2 (fr) * 2013-11-04 2015-05-07 Dow Agrosciences Llc Locus optimaux de soja
EP3960856A1 (fr) * 2013-11-13 2022-03-02 Children's Medical Center Corporation Régulation de l'expression génique médiée par la nucléase
EP3757116A1 (fr) 2013-12-09 2020-12-30 Sangamo Therapeutics, Inc. Procédés et compositions pour l'ingénierie génomique
BR112018008519A2 (pt) 2015-10-28 2018-11-06 Sangamo Therapeutics Inc construtos específicos de fígado, cassetes de expressão de fator viii e métodos de uso dos mesmos
AU2016369490C1 (en) 2015-12-18 2021-12-23 Sangamo Therapeutics, Inc. Targeted disruption of the T cell receptor
CN109069568B (zh) 2016-02-02 2023-07-07 桑格摩生物治疗股份有限公司 用于连接dna结合结构域和切割结构域的组合物
AU2017315414B2 (en) 2016-08-24 2024-02-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100113575A1 (en) * 2007-03-27 2010-05-06 Ayesha Sitlani Method for detecting autoprocessed, secreted pcsk9
US20140356958A1 (en) * 2012-12-17 2014-12-04 President And Fellows Of Harvard College RNA-Guided Human Genome Engineering
US20160153005A1 (en) * 2013-06-17 2016-06-02 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
US20150128309A1 (en) * 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal Maize Loci
US20150335708A1 (en) * 2014-05-08 2015-11-26 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
US20150329875A1 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
DING ET AL.: "Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing", CIRC RES., vol. 115, no. 5, 2014, pages 488 - 92, XP055428398 *
DURAL ET AL.: "Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells", NUCLEIC ACIDS RES., vol. 33, no. 18, 2005, pages 5978 - 90, XP002511419 *
WANG ET AL.: "CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report", ARTERIOSCLER THROMB VASC BIOL., vol. 36, no. 5, 2016, pages 783 - 6, XP055385148, [retrieved on 20160303] *

Also Published As

Publication number Publication date
EP3509645A2 (fr) 2019-07-17
AU2017324462A1 (en) 2019-03-14
CA3035534A1 (fr) 2018-03-15
US10960085B2 (en) 2021-03-30
AU2017324462B2 (en) 2024-03-21
WO2018049009A2 (fr) 2018-03-15
JP2019531068A (ja) 2019-10-31
EP3509645A4 (fr) 2020-06-17
US20210060179A1 (en) 2021-03-04
JP7256739B2 (ja) 2023-04-12
US20180064827A1 (en) 2018-03-08
JP2023082133A (ja) 2023-06-13

Similar Documents

Publication Publication Date Title
WO2018049009A3 (fr) Modulation des gènes du foie
WO2019006471A3 (fr) Nouveaux enzymes de ciblage d'arn crispr, systèmes et utilisations associés
AU2018320870A1 (en) RNA targeting methods and compositions
ZA201805009B (en) Stable inoculant compositions and methods for producing same
AU2017220012B2 (en) Nucleic acid carriers and therapeutic methods of use
EP4219532A3 (fr) Compositions et procédés pour une thérapie génique transitoire à stabilité améliorée
MX2018016038A (es) Compuestos y metodos para modular la funcion del acido ribonucleico (arn).
EP3585424A4 (fr) Protéines de liaison multi-spécifiques ciblant caix, ano1, la mésothéline, trop2, cea ou la claudine-18.2
WO2018109170A3 (fr) Anticorps il-11ra
WO2017075478A3 (fr) Compositions et méthodes d'évaluation et de modulation des réponses immunitaires à l'aide de signatures génétiques de cellules immunitaires
MX2015003874A (es) Modulacion de ire1.
WO2018109174A3 (fr) Anticorps anti-il-11
EP3604535A3 (fr) Procédés et compositions utilisables pour lutter contre les mauvaises herbes
EA201591474A1 (ru) Модуляторы метил-модифицирующих ферментов, композиции и их применение
WO2017093804A3 (fr) Matériaux et méthodes de traitement d'une déficience en antitrypsine alpha-1
PH12015501720A1 (en) Modulators of methyl modifying enzymes, compositions and uses thereof
EP4218411A3 (fr) Stabilisation et isolement d'acides nucléiques extracellulaires
MX2022000053A (es) Moduladores de interaccion de sestrina-gator2 y sus usos.
MX2020004043A (es) Composiciones y métodos para la edición génica de hemofilia a.
WO2015191951A3 (fr) Modulation d'activité du complément
MX2018016057A (es) Composiciones y metodos para tratar enfermedades cardiovasculares.
WO2017132746A8 (fr) Inhibiteurs 53bp1
PH12018502233A1 (en) ErbB INHIBITORS AND USES THEREOF
WO2015161170A3 (fr) Compositions et procédés de modulation de l'épissage du smn2 chez un patient
WO2019051327A3 (fr) Agents de modulation des fonctions de la bêta-caténine et méthodes associées

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 17849517

Country of ref document: EP

Kind code of ref document: A2

ENP Entry into the national phase

Ref document number: 3035534

Country of ref document: CA

ENP Entry into the national phase

Ref document number: 2019512750

Country of ref document: JP

Kind code of ref document: A

NENP Non-entry into the national phase

Ref country code: DE

ENP Entry into the national phase

Ref document number: 2017324462

Country of ref document: AU

Date of ref document: 20170907

Kind code of ref document: A

ENP Entry into the national phase

Ref document number: 2017849517

Country of ref document: EP

Effective date: 20190408